Prosensa Natural History Study (Pro DMD Study)

What is this study about?

This study is to:

Determine the muscle strength and function of subjects with (DMD) and its evolution with time
Assess the quality of life of DMD patients
Measure biomarkers (some proteins found in the blood and urine that may be linked to disease progression)
There is no medication being tested in this study

What do we do in this study?

During a study visit, patients are asked to:

Perform some muscle testing with a physical therapist to see how well they can:
Walk, run, jump, climb stairs
Move their arms, legs, hands, fingers
Breathe in a machine
Answer surveys about quality of life and how they think they can perform daily tasks
Provide blood and urine samples

Who can participate?

DMD patient with any type of mutation (exon deletion, du-plication, stop codon…)
Age range at enrolment: between 3 and 18 years
It is acceptable to take medications during the study. Only investigational drug products are excluded
If a drug study becomes available, patients will be able to leave the natural history study and move into the drug study
250 patients will be enrolled in 16 hospitals in 10 countries